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Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.

AcelRx Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on the development and commercialization of therapies for the treatment of acute pain. Its lead product candidate is DSUVIA and DZUVEO, a 30 mcg sufentanil sublingual tablet for the treatment of moderate-to-severe acute pain. The company develops ZALVISO, a patient-controlled analgesia system that allows hospital patients with moderate-to-severe acute pain to self-dose with sufentanil sublingual tablets to manage their pain; ARX-02, which is in Phase 2 clinical trial for the treatment of cancer breakthrough pain in opioid-tolerant patients; and ARX-03, which is in Phase 2 clinical trial for the treatment of procedural anxiety and acute pain. Its product candidates also include Niyad, a regional anticoagulant for the extracorporeal circuit; and LTX-608, a nafamostat formulation for direct IV infusion being explored as an investigational product for antiviral treatment of COVID, acute respiratory distress syndrome, disseminated intravascular coagulation, and acute pancreatitis. The company also develops Fedsyra, an ephedrine pre-filled syringe that contains 10 ml of a solution of 3 mg/ml ephedrine hydrochloride for injection; and phenylephrine, a phenylephrine pre-filled syringe, which contains 10 ml of a solution of 50 mcg/ml phenylephrine for injection. The company was formerly known as SuRx, Inc. and changed its name to AcelRx Pharmaceuticals, Inc. in August 2006. AcelRx Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Hayward, California.

Helix BioPharma Corp. operates as an immune-oncology company in Canada. It is developing L-DOS47 for the treatment of non-small cell lung cancer; and V-DOS47 that targets the vascular epithelial growth factor receptor 2. Helix BioPharma Corp. has collaboration agreements with Moffitt Cancer Center to investigate the pharmacodynamics of L-DOS47 and determine the benefits of combining L-DOS47 with immune checkpoint inhibitors; and with University Hospital Tuebingen to assess the therapeutic response of L-DOS47 in cancer models expressing CEACAM6, with advanced preclinical metabolic imaging. The company is headquartered in Toronto, Canada.

Cassava Sciences, Inc., a clinical stage biotechnology company, develops drugs for neurodegenerative diseases. Its lead therapeutic product candidate is simufilam, a small molecule drug, which is completed Phase 2 clinical trial; and investigational diagnostic product candidate is SavaDx, a blood-based biomarker/diagnostic to detect Alzheimer's disease. The company was formerly known as Pain Therapeutics, Inc. and changed its name to Cassava Sciences, Inc. in March 2019. Cassava Sciences, Inc. was incorporated in 1998 and is based in Austin, Texas.

Savara Inc., formerly Mast Therapeutics, Inc., is a clinical-stage pharmaceutical company. The Company is focused on the development and commercialization of novel therapies for the treatment of patients with rare respiratory diseases. Its pipeline includes AeroVanc, Molgradex and AIR001. AeroVanc is an inhaled formulation of vancomycin, which the Company is developing for the treatment of persistent methicillin-resistant Staphylococcus aureus, lung infection in cystic fibrosis patients. Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor. It is developing Molgradex for the treatment of autoimmune pulmonary alveolar proteinosis, a rare lung disease. AIR001 is a sodium nitrite solution for inhalation via nebulization. AIR001 is in Phase II clinical development for the treatment of heart failure with preserved ejection fraction, also known as diastolic heart failure or heart failure with preserved systolic function.